The testing of new medicines can be a lengthy and arduous process, delaying access to promising innovative treatments. Today’s video features a TED talk from bio researcher Geraldine Hamilton, who creates ‘organs-on-a-chip’ – engineering a ‘home away from home’ for the cells which more accurately recreates the dynamic environment in which they exist in the body.

Watch to find out how the chips are created and how they are being utilised for the testing of new medicines, including in the burgeoning field of personalised medicine.

The biotech sector continues to strengthen and evolve across the globe, developing innovative treatments through new routes. In the UK, our world-class research base has long provided the strong foundations for a thriving ecosystem. Here, David Campbell, Chief Executive Officer, Magnus Life Science, details their ‘spin in’ approach to nurturing the ground-breaking biomedical research undertaken in the UK.

The biotech world is facing exciting times:

There is a major resurgence in the UK biotechnology community

There is more investment than ever going into driving innovative, new biotech science

We are at an inflection point in science and are on the cusp of seeing new, exciting sources of treatment making their mark on society and having a significant influence on the way that we treat patients and potentially cure disease

Like many executives involved in this industry, I am extremely excited to be involved at this particular point in time but I am also a little worried. Whilst the environment that we operate in has undoubtedly changed, has our business model kept pace?

Funding sources – and I would contend their attitude to risk – remain the same. However, the model of sourcing innovation from our world-leading universities is today handicapped by systematic failings in our technology transfer approach. So what do we do? Like the scientists that we employ to drive our innovation, perhaps it’s time to experiment.

At Magnus we passionately believe that universities remain the singularly most important source of innovation and are the fuel for growth of our biopharmaceutical industry. We also believe that the NHS remains one of the best systems in the world in which to test and further develop the next generation of medicines. The sources of funding can certainly remain the same – but how we combine these three factors cannot.

When most people think about commercialising ground-breaking university research, the model they’re likely to have in mind is the ‘spin-out’. It’s an established route and the go-to model for our academics and universities. However, at Magnus Life Science, we believe the spin-out model has significant shortcomings and that much research would be better served – and ultimately have more commercial impact – if a ‘spin-in’ model was used instead.

A spin-in, as we see it, involves taking an entire company and putting it within a university. Innovation needs to be nurtured. It’s the opposite to the ‘chicks out of the nest’ approach of the spin-out. It also has advantages over the use of incubators which do provide a supportive environment but still fall short of providing the full infrastructure and culture available to a business truly embedded in a university.

With this approach, science is nurtured rather than being sent out into the wilderness. It’s a warmer environment, with many of the barriers between the academic and commercial worlds broken down, and one we think will allow us to progress our programmes more effectively.

The UK is an ideal place to adopt this new approach to translational science. As mentioned above, we have some of the world’s leading academics in some of the world’s leading universities. Second, we have a health care system that is willing to experiment and provide early access to potentially innovative new medicines. Finally, in the UK and in Europe, we have access to some of the largest pots of venture capital managed by some of the most knowledgeable and experienced investors and entrepreneurs. These are not solely sector specialists but also investors who can be attracted to life sciences from other high growth areas.

The model that we have developed at Magnus aims to bring all of these key stakeholders together. Through an industrial collaboration, the Company’s operational and R&D teams are located (“spun-in”) within University College London with renowned experts who are practising clinicians working closely with an experienced management team to take the academic, commercial and clinical lead on projects.

There are challenges to the model, of course. At the core of the model has to be trust. Trust that the spin-in company will not abuse its privileged position. Trust that the university and their staff will respect the commercial sensitivity of the work being performed and help protect the valuable IP being generated.

The university has to be incentivised but there are ways and means of achieving this through fee for service, equity based models and even opportunities to access additional funds for the university through the UK Quality-related Research (QR) funding process.

There is also a limit to the size a company could reach as a spin-in – perhaps 200 to 300 people – and a fine balance between academic research and freedom of creativity vs commercial pressures to deliver.

But for us, we believe the spin-in model will be core to our success and I am confident that our progress will be seen as an example and help lead to more excellent research having the commercial and societal impact it deserves.

Another week, another key deal for our sector. Today Heptares Therapeutics announced it is to be acquired by Japanese biopharmaceutical company, Sosei,for up to $400 million. This significant dealsecures significant investment into the company well into the future. I understand that Heptares will be run as a largely autonomous wholly-owned subsidiary and its operations at Welwyn will be staying and will become a major global drug discovery and development hub for Sosei here in the UK. Congratulations to Malcolm Weir and the Heptares team.

A boost for industrial biotechnology last week as Vince Cable announced the latest winners of Industrial Biotechnology Catalyst funding, worth a combined £20 million. Cable made the announcement on a visit to Ingenza in Edinburgh, one of the many BIA members to win funding in this latest round. The 23 winning projects cover a wide range of applications – from developing new antibiotics to making biofuel from household waste – showcasing the huge spectrum of potential benefits from this emerging and rapidly developing field. So congratulations to all 12 BIA members who received funding – I look forward to hearing more on the projects as they develop.

On the topic of catalysts, I’m looking forward to our CEO dinner in Oxford later this week where we’ll be discussing the past, present and future of the Biomedical Catalyst. The BMC has been one of the key focuses of BIA lobbying and advocacy work and has proved itself to be a great success from the current government. Since the scheme was launched in 2012 over £200 million has been invested through the scheme and it has leveraged a further £100 million in industry co-commitment. Over 25 BIA member companies have won funding from the scheme, including Modern Biosciences who recently wrote us a blog on the vital role it has played in their journey. As we head into an election year where the future of the scheme will be under the consideration by whoever holds the keys to power after 7 May, I’m sure there will be plenty of lively discussion over dinner on Wednesday – I’ll report back next week with any highlights. The following morning we’ll also be holding our Breakfast with the BIA – the second of the investor-ready road show events. I hope to see some of you there.

On the regulatory front, last week the BIA inputted, together with EuropaBio, to an important consultation by the EMA on proposals on how the transparency requirements of the EU Clinical Trials Regulation will be applied to data and information in clinical trial applications which will be stored in the new EU database. In our response, the BIA has supported the need for a balanced approach to be taken that respects both the rights of patients and the public to be able to access information held on the EU database concerning ongoing clinical trials, as well as the needs of developers and researchers to protect their investments and cutting edge research and development of new innovative medicines. We’ll be taking forward our engagement in the coming weeks and will report back with any developments.

We are currently drafting the BIA’s response to a review by Dame Anne Dowling on Business-University Research Collaborations. It’s not long since the House of Commons undertook a similar inquiry (and you can see our response to that here), but Science Minister Greg Clark has particularly asked for the Dowling Review to look at case studies of long-term collaborations and successful practices (in the UK and elsewhere and in any sector) that have led to fruitful partnerships. The whole aim is to identify the barriers to collaboration and make recommendations for practices that can be replicated more widely, including areas where the future government can give effective support, perhaps through schemes or incentives. If you have experience of a long-term collaboration you’d like to share some thoughts on the topic and input into our response, we’d like to hear from you – please get in touch with Zoe.

The BIA has been approached to facilitate a chapter in the upcoming edition of European Biotechnology, which would also feature 20 to 25 UK biotech company profiles. If you think you would be interested in contributing, please get in touch.

This week’s video features an interview with Professor Dame Sally Davies, the UK’s Chief Medical Officer, from PharmaTelevision. In the clip, Dame Sally describes why the UK’s biotech sector is a key part of the ecosystem, not only for producing innovations that make a difference for patients but also for the economy and growth.

Dame Sally also explains the background to the controversial vote in the House of Commons for regulations to allow mitochondrial transfer and her role on the committee for the Longitude Prize which is looking to help tackle the global problem of antibiotic resistance.

The current gold standard across most regulators, including the New York State Department of Health Oncology guidelines, ISO 15189 and CLIA guidelines, are focused on use of patient specimens for verification and validation activities. Is the availability and reproducibility of alternative reference materials improved to the point that other reference material sources could be considered? Hannah Murfet of Horizon Discovery evaluates the potential of cell line derived reference materials.

The use of reference materials, whether derived from patient specimens or otherwise is clear for demonstrating validity across the clinical supply chain. From drug discovery to detection mechanisms to the clinical laboratory, reference materials are essential to determine key performance characteristics such as reproducibility and sensitivity. Patient derived samples often have the limitation of a supply limit. With a lack of sustainability comes a lack of a permanent benchmark, and as patient derived reference materials are depleted, an alternative may be hard to source, introducing further variability and uncertainty.

Through limited availability and distribution, use of patient specimens introduces variability between hospitals and even the same hospital within a period of time. Cell line based reference materials can be produced from immortalised line that are sustainable and able to be modified to meet the clinical need through targeted genetic engineering, giving the potential to represent even the clinically rare patient specimens. Developments in genetic engineering and use of similar matrices to patient specimens allow greater potential for cell line reference material used for pre-analytical aspects of the workflow such as extraction and sample preparation.

Cell line derived reference materials can represent much closer models to patient specimens through the format matrices, such as FFPE or on-slide reference materials. Other reference material providers are emerging for new technologies such synthetic constructs, however further work is required to determine acceptable use of these for validation. Meanwhile valuable efforts such as the Genome in a Bottle Consortium aim to characterise human genome sequences for reference materials through repeated testing and community distribution. In spite of this, there is still a perception over a lack of available reference materials, including those available for proficiency testing of genetic tests (Congressional Research Service).

There is no doubt that reference materials are required for developing the essential case for clinical utility and validity across the clinical supply chain. Development in the matrices such as FFPE or even serum substrates may further enhance the patient relevance of cell line derived materials to a point where they may be seen as equivalent. Availability of defined cell line based from genetic engineering can improve the availability of reference materials, so perhaps it is time to review the focus on patient specimens. Perhaps it is time to consider the utility of cell-line derived reference materials and their value proposition of long term sustainability in validation and verification in the clinical supply chain.

Last week the Mayor of London led a delegation to the US East Coast, showcasing UK life sciences in Boston and New York. Sarah Haywood, Chief Operating Officer, MedCity, provides some of her reflections from the trip and examines how the UK scales up to the likes of Boston – the Mecca of the life sciences sector.

There are lots of ways to keep warm when the temperature is way below freezing. In New York last week as part of the Mayor of London’s delegation to the US East Coast, Reza Razavi of King’s Health Partners and I took the athletic option of hauling our suitcases through the upper edges of Manhattan to visit the Harlem Biospace. It was well worth the effort to see what is being achieved there – seventeen very young companies in a real incubator that was kitted out for $600k. Our reaction – why can’t we do this?

Despite the thick snow that caused some hasty schedule rearrangements (Harvard was closed, MIT partially so), being part of the Mayor’s delegation in Boston and New York has been inspiring and energising. We were greeted everywhere with genuine enthusiasm for the UK and recognition of our life sciences excellence. Companies want to carry out clinical trials and develop bases here as they bring products to the US market. There is significant collaboration with UK academic centres and an appetite for more.

Boston, of course, is the Mecca of the life sciences sector – the place that famously has the highest concentration of companies within a two mile square radius of anywhere on earth. It’s instructive to meet the community there to find out what we can learn, but also see how finely matched we are; in many ways, I would argue, the UK has the edge.

For Boris Johnson, who really gets life sciences and is actively supporting the sector, the big message for his US visit was that the UK has a massively innovative culture that presents huge opportunities for industry and investors alike. For life sciences, that is underpinned by some of the world’s best universities and healthcare centres, and, particularly in London, a hugely diverse population served by a single, integrated health system. We are already attracting serious investment from the US, not least from Gilead Sciences and Boston-based health tech company Mobiquity, who both announced new $20m HQs in London last week.

We know we have challenges – not enough incubator and grow on space for all the emerging and growing companies that the sector is producing; and access to investment for life sciences in the UK does not yet match what can be sourced in the US. These are all challenges that can be tackled, especially when we have the political backing we are now lucky enough to enjoy.

For me, the highlight of the trip was the opportunity to meet the great serial entrepreneur and academic powerhouse Robert Langer. His advice for the UK? Culture change takes time but persevere – you will get there. You need role models – individuals and companies that aspiring entrepreneurs can look at and think ‘they did it, and so can I’. And mixing disciplines – particularly engineers and bioscientists – is where the really important and successful innovations are created.

The UK life sciences sector ticks all those boxes and we can already see the results. It’s not too ambitious to envisage a time when the Boston mayor leads a life sciences delegation to London, to benefit from our wisdom and our weather.

Following on from last week’s number of good news stories for biotech investment, the UK ecosystem saw a further boost as two US healthcare companies – Gilead Sciences and Mobiquity – announced their plans to move to London. The announcements were made as part of Boris Johnson’s mission to the US, and will generate an additional £26 million investment in the city. Keep an eye on our blog as we’ll be posting a round up from the mission from MedCity later this week. Meanwhile, Harriet Fear from One Nucleus was flying the flag for United Life Sciences in India last week during an oncology-focused trade mission to Bangalore and Mumbai.

In the world of industrial biotechnology (IB) and synthetic biology, BIA member Synthace announced a funding boost of £2.2 million to expand their bioengineering automation and further develop ‘Antha’, an open-source language that enables rapid automation of reproducible biological lab processes in potentially diverse fields. Synthace, Ingenza, Algenuity, CHAIN Biotech and other BIA members took part in the IB Leadership Forum’s IB Showcase event last week, which was by all accounts a great couple of days (see #IB15 to catch up on tweets). It’s great news that CHAIN Biotech won the IBLF Award for Most Promising SME. And it’s encouraging to see cross-disciplinarity, with industry chairs from several Leadership Councils including IB, synthetic biology and the Medicines Manufacturing Industry Partnership (MMIP) discussing the bioeconomy. If an engineering-based approach to life science problem-solving could be of interest to your company, our Synthetic Biology Advisory Committee are keen to hear from the wider BIA membership so do get in touch.

More good news came in the form of Imperial College London’s plans to invest in a new facility at the Babraham Research Campus, which will support spin-out and scale-up companies and maximise the impact of research from universities. BIA member Abzena has been announced as the anchor tenant at the new site, which will enable scientists and support teams from its subsidiary businesses, Antitope and PolyTherics, to be housed in the same building and in a cutting-edge R&D environment.

On the topic of world-class lab environments, last week’s Panorama ‘Can you cure my cancer?’ included footage inside Institute of Cancer Research (ICR)’s ‘mouse hospital’, where researchers carry out pre-clinical testing of candidate cancer drugs. It’s well worth a watch as a great example of how organisations like the ICR are helping to make animal research better understood as part of wider public dialogue about medical research and scientific progress.

The Royal Society, the British Academy for the Humanities and Social Sciences, the Royal Academy of Engineering and the Academy of Medical Sciences (AMS) last week launched a pre- general election report ‘Building a stronger future: Research, Innovation and growth’. Page 7 rightly mentions the need for effective policies to support industrial strategy, including policies on access to finance. I was delighted to be able to host a table at the Royal Society ‘Labs to Riches’ evening last week, it was fantastic to see the vibrant network of UK science celebrating and endorsing translational success.

The following day was the AMS’s FORUM Annual Lecture, featuring a keynote from Life Sciences Minister George Freeman MP (busy as ever!) and a panel discussion with the three Life Sciences Champions Sir John Bell, Chris Brinsmead and John Jeans. AZ’s Mene Pangalos brought up the subject of the need for funding to support translation and to retain that activity here in the UK, and I’m pleased that the panellists recognise this; Brinsmead suggested we should be more courageous, we must attract investment capital, and the Treasury should look at tax advantages; while Jeans agreed we need to build up a UK investor base and Bell noted that the Minister is thinking hard about the value of long term patient capital.

We also always have to be patient about real world data and I was glad to see our Charity partner from 2013, the Cystic Fibrosis Trust, comment on new England and Wales data from the Cystic Fibrosis (CF) Registry which have revealed that BIA member company Vertex’s drug Kalydeco, rapidly adopted by the NHS in the last two years, has significantly increased patient lung function. This reinforces calls for it to be made available to further patients with rare CF genotypes.

Also a quick note on funding opportunities as EuropaBio’s Most Innovative European Biotech SME Award is open for applications – this is taking place earlier than usual this year, and applications will close on 6 April. The 2013 winner was BIA member PsiOxus Therapeutics, so I’d encourage you to apply if you can and fly the flag for innovation in UK biotech.

Finally, I just wanted to flag to you that we are about to commence a BIA membership survey. Members will receive an email from Research by Design with the survey appended – I really encourage you to complete it so we can get your feedback on how we best meet your needs as your sector trade association.

This week’s member video from the Biotechnology and Biological Sciences Research Council (BBSRC) is the last in a series of three videos presenting technological, economic and public impacts of BBSRC-funded research over the past 20 years, including revolutionary advances in DNA sequencing, atomic force microscopy for nano-scale visualisation, development of biofuels and vaccines, and much more.

Open Innovation continues to make its mark on the bioscience sector. It has brought us increased levels of well-established forms of collaboration, such as university-business interactions – and it has heralded many new methods such as crowd-sourcing and open data initiatives. Following Stevenage Bioscience Catalyst‘s recent Open Innovation Summit, Clare O’Neill of Original Ventures and Miranda Knaggs of SBC give us the inside track on the main conclusions.

It was Berkeley academic Henry Chesbrough who first coined the term ‘open innovation’ to describe any external collaboration. This was a useful concept in the ongoing fight against Not Invented Here Syndrome – a disease that has prevented many organisations from spotting new opportunities and remaining competitive.

Stevenage Bioscience Catalyst (SBC), the UK’s first open innovation bioscience campus, recently held its third annual Open Innovation (OI) Summit, an opportunity for leading lights in the bioscience sector to share their real life OI experiences and insight.

One of the main themes arising from discussions was the increased understanding of OI. It should be seen as a spectrum, rather than an open/closed state – each organisation or person can select what’s best for them, case-by-case. In other words, you simply share what you want and you keep the rest. A safe environment for OI is one where the participants have agreed on the rules of engagement for each step of the collaboration.

Benefits for all

Small companies and academic researchers rarely get access to feedback from big pharma, to help them define their commercial goals and shape their R&D strategy in exactly the right way to attract the next stage of investment.

OI improves this state of affairs, through the growing number of open innovation challenges where big pharma publicly invite anyone to submit ideas or solutions to a defined issue. These challenges often allow for confidential disclosure and subsequent agreements on IP rights.

SBC was created to enable more of this pharma feedback on site at the GSK Stevenage R&D campus, exploiting the proximity of bioscience tenants with GSK’s research groups, investment experts, and facilities – but without any obligation by tenants to interact or do any kind of deal with GSK. Academic groups across the UK who have taken part in SBC’s recent open innovation challenge on unmet needs in neuro-degenerative disease, and the SBC Discover Assist programme, praised the early access to big pharma feedback as a real plus, allowing them to improve research outlines and business plans.

Another OI advantage comes from the crowd. Very early-stage projects often languish in the infamous Funding Gap, where investors fear to tread. Because of the relative risk, securing the first £50k for a new venture is usually more difficult than a later-stage £1m. However each year brings more successful examples of crowd-funding being used to drive good science through this gap and onto bigger things.

Coffee machines and bumper cars

The importance of random chats by the coffee machine is far more widely appreciated now as an enabler of innovation. Scaling that up, SBC uses what CEO Martino Picardo calls the ‘bumper car’ model of innovation: biotechs, big pharma, academics, start-ups and seasoned business experts can easily and regularly bump into each other on campus, stimulating even more cross-talk to exchange knowledge and expert opinion.

OI can be used to bring together ‘the unusual suspects’ from across disciplines and sectors, and to increase the knowledge and insight available to the whole sector, raising the quality bar for everyone. Ultimately we are all patients, and any new methods of bringing ideas and expertise together benefits us all.

As 2015 continues apace we have one final reflection on last year from the perspective of our supported charity for 2014, Fight for Sight, as Chief Executive Michele Acton gives an overview of what the partnership meant to them. It has been a pleasure!

2014 was quite a year for Fight for Sight and for eye research. Being the BIA charity gave us a huge boost. Our partnership kicked off to a great start with the charity being asked to present at the joint BIA and Association of Medical Research Charities (AMRC) Parliamentary Reception which highlighted how organisations from all sectors can work together to maximise benefits for patients.

Michele Acton speaking at our parliamentary event with AMRC

Earlier that same day we were delighted that BIA member, Syncona LLP, announced a £12 million investment in Nightstar, a spin-out from the University of Oxford, focusing on developing and commercialising therapies for inherited eye diseases with the first program being a gene therapy for choroideremia. Funding from the Tommy Salisbury Choroideremia Fund at Fight for Sight had led to the world’s first trial for a treatment for choroideremia. This Phase 1 trial showed promising results underpinning the new investment.

Our work to address inherited eye diseases was also the subject of a passionate talk at the annual BIA Gala Dinner at the Natural History Museum. Golf professional Mark Roberts, father to nine year old Rose who has Stargardt’s, spoke on behalf of Fight for Sight about the impact of the condition. BIA members responded so generously and raised a record breaking £31,000 for eye research.

Fight for Sight funds research with the aim of preventing and developing treatments for a wide range of different eye diseases and conditions. Towards the end of 2013 the Sight Loss and Vision Priority Setting Partnership was published. This highlights the key questions that patients and eye health professionals want answered by research. It helps us ensure that our funding is focussed on what is most important to those with experience of sight loss. During 2014 we worked with the NIHR Horizon Scanning team to develop a report on potential new treatments that are being developed across the world for inherited eye diseases. These were reported here in a BIA blog.

Emma Salisbury speaking at the UK Bioscience Forum

At the UK Bioscience Forum in the autumn, we listened to a great speech by our Patron, Lord Drayson. We followed this with a tag team presentation which showed how inspirational individuals can work with charities to leverage further funding to take research from bench to bedside, using choroideremia as a case study. Those of you who attended will no doubt remember the inspirational Emma Salisbury whose fundraising was integral to the initial funding. As a mother of a son with choroideremia Emma is a passionate fundraiser who has just been named Tesco Achieving Mum of the Year.

Steve Bates and Michele Acton completing the Carrots NightWalk

Our partnership throughout 2014 has meant so much. It has given us the opportunity of raising the profile of our work and the need for eye research amongst BIA members and beyond. We have built relationships that will continue long after the year has ended. We are so grateful for everybody’s generosity in raising funds and in going the extra mile in offering their advice and time…..this includes a big thank you to Steve Bates who went the extra 15 miles for us by being sponsored to walk in our Carrots NightWalk. We are very sorry to say goodbye as the charity of the year but wanted to thank you for giving us such a great platform to build upon. Please do follow our progress at www.fightforsight.org.uk.